WO2020257590A8 - Production de vecteurs utilisant une origine de réplication de phage - Google Patents

Production de vecteurs utilisant une origine de réplication de phage Download PDF

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Publication number
WO2020257590A8
WO2020257590A8 PCT/US2020/038651 US2020038651W WO2020257590A8 WO 2020257590 A8 WO2020257590 A8 WO 2020257590A8 US 2020038651 W US2020038651 W US 2020038651W WO 2020257590 A8 WO2020257590 A8 WO 2020257590A8
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WO
WIPO (PCT)
Prior art keywords
nucleic acid
replication
circular nucleic
production
phage origin
Prior art date
Application number
PCT/US2020/038651
Other languages
English (en)
Other versions
WO2020257590A1 (fr
Inventor
Richard Jude Samulski
Lester SUAREZ
Original Assignee
Asklepios Biopharmaceutical, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Asklepios Biopharmaceutical, Inc. filed Critical Asklepios Biopharmaceutical, Inc.
Priority to US17/620,557 priority Critical patent/US20220243212A1/en
Priority to AU2020295507A priority patent/AU2020295507A1/en
Priority to JP2021576043A priority patent/JP2022537410A/ja
Priority to EP20827801.0A priority patent/EP3987043A4/fr
Priority to CN202080059131.0A priority patent/CN114269938A/zh
Priority to CA3144364A priority patent/CA3144364A1/fr
Publication of WO2020257590A1 publication Critical patent/WO2020257590A1/fr
Publication of WO2020257590A8 publication Critical patent/WO2020257590A8/fr

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2795/00—Bacteriophages
    • C12N2795/00011—Details
    • C12N2795/14011—Details ssDNA Bacteriophages
    • C12N2795/14111—Inoviridae
    • C12N2795/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2820/00—Vectors comprising a special origin of replication system
    • C12N2820/60—Vectors comprising a special origin of replication system from viruses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La présente invention concerne un procédé de fabrication de vecteurs d'acide nucléique circulaires contenant un transgène comprenant les étapes consistant à : (A) mettre en contact un système hôte avec un modèle, le modèle comprenant au moins un site de clivage flanquant, et (i) au moins une origine de réplication de phage (ORI); (ii) au moins une répétition terminale (TR), et; (iii) une séquence de promoteur fonctionnellement liée à un transgène; (b) incuber le système hôte pendant une durée suffisante pour permettre à la réplication de se produire, pour ainsi conduire à une production d'acide nucléique circulaire; et (c) récupérer la production d'acide nucléique circulaire, l'acide nucléique circulaire s'auto-hybridant.
PCT/US2020/038651 2019-06-21 2020-06-19 Production de vecteurs utilisant une origine de réplication de phage WO2020257590A1 (fr)

Priority Applications (6)

Application Number Priority Date Filing Date Title
US17/620,557 US20220243212A1 (en) 2019-06-21 2020-06-19 Production of vectors using phage origin of replication
AU2020295507A AU2020295507A1 (en) 2019-06-21 2020-06-19 Production of vectors using phage origin of replication
JP2021576043A JP2022537410A (ja) 2019-06-21 2020-06-19 ファージ複製起点を用いたベクターの産生
EP20827801.0A EP3987043A4 (fr) 2019-06-21 2020-06-19 Production de vecteurs utilisant une origine de réplication de phage
CN202080059131.0A CN114269938A (zh) 2019-06-21 2020-06-19 使用噬菌体复制起点生产载体
CA3144364A CA3144364A1 (fr) 2019-06-21 2020-06-19 Production de vecteurs utilisant une origine de replication de phage

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962864689P 2019-06-21 2019-06-21
US62/864,689 2019-06-21

Publications (2)

Publication Number Publication Date
WO2020257590A1 WO2020257590A1 (fr) 2020-12-24
WO2020257590A8 true WO2020257590A8 (fr) 2021-08-19

Family

ID=74037424

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2020/038651 WO2020257590A1 (fr) 2019-06-21 2020-06-19 Production de vecteurs utilisant une origine de réplication de phage

Country Status (7)

Country Link
US (1) US20220243212A1 (fr)
EP (1) EP3987043A4 (fr)
JP (1) JP2022537410A (fr)
CN (1) CN114269938A (fr)
AU (1) AU2020295507A1 (fr)
CA (1) CA3144364A1 (fr)
WO (1) WO2020257590A1 (fr)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
WO2022156188A1 (fr) * 2021-01-22 2022-07-28 Nanjing GenScript Biotech Co., Ltd. Procédé de production d'une séquence d'adn cible et vecteur de clonage
CN118679248A (zh) * 2021-09-27 2024-09-20 阿尔德弗龙有限责任公司 环状dna载体的合成产生
WO2024030432A1 (fr) * 2022-08-01 2024-02-08 Gensaic, Inc. Particules dérivées de phages thérapeutiques

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU773277B2 (en) * 1996-09-20 2004-05-20 Cold Spring Harbor Laboratory Viral vectors and their uses
WO2002057447A2 (fr) * 2001-01-19 2002-07-25 Genetica, Inc. Methodes et reactifs pour amplification et manipulation de sequences vecteurs et cibles d'acide nucleique
CA2332186A1 (fr) * 2001-02-08 2002-08-08 Her Majesty In Right Of Canada As Represented By The Minister Of Agricul Ture And Agri-Food Canada Systemes de ciblage in vivo de genes de replication
WO2002062986A2 (fr) * 2001-02-08 2002-08-15 Her Majesty In Right Of Canada As Represented By The Minister Of Agriculture And Agrifood Canada Ciblage genique in vivo replicatif
EP2500434A1 (fr) * 2011-03-12 2012-09-19 Association Institut de Myologie Vecteurs AAV sans capside, compositions et procédés pour la production des vecteurs et la thérapie génique
US20130274129A1 (en) * 2012-04-04 2013-10-17 Geneart Ag Tal-effector assembly platform, customized services, kits and assays
CA3104113A1 (fr) * 2018-06-22 2019-12-26 Asklepios Biopharmaceutical, Inc. Vecteurs pour l'administration de genes qui persistent dans les cellules

Also Published As

Publication number Publication date
EP3987043A1 (fr) 2022-04-27
CN114269938A (zh) 2022-04-01
US20220243212A1 (en) 2022-08-04
WO2020257590A1 (fr) 2020-12-24
EP3987043A4 (fr) 2023-07-19
JP2022537410A (ja) 2022-08-25
CA3144364A1 (fr) 2020-12-24
AU2020295507A1 (en) 2022-02-17

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